Martina's introduction to the ISPOR Glasgow workshop on orphan drugs.

1. Sustainable funding and fair pricing for
orphan drugs. What are the solutions?
ISPOR European Congress 2017

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Introduction
• Ongoing debate on sustainable funding of
orphan drugs
• The evidence in support of paying a premium
for orphan drugs is mixed
• However, many countries in Europe provide access
• The aim of this workshop is to discuss options
• To make the funding of valuable orphan drugs
sustainable for healthcare systems
• To provide a ‘fair’ reward to manufacturers investing in
areas of high unmet need

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Structure of the workshop
• Martina Garau
Rate of HTA approval/reimbursement of orphan drugs in EU
• Saskia Knies
Insights and learnings from the Dutch approach
• Mike Drummond
New method to adjust the cost effectiveness threshold to
reflect the different population sizes

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Definitions
• Orphan drugs prevalence: no more than
5 in 10,000 patients (EMA eligible
criteria)
• Ultra-orphan drugs prevalence: less than
1 in 50,000 (NICE, 2004)

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Voting
1. Do you agree that there should be a higher cost
effectiveness threshold for orphan drugs compared to
that used to appraise treatments for common conditions?
2. If you agree, on which basis the threshold should be
adjusted?
3. If adjustments to the threshold are possible, should
decision makers distinguish between orphans and ultra-
orphans?
4. Do you agree that the evidence requirements for orphan
drugs should be different (or less stringent) than those
expected for common conditions?

6. Comparing HTA approval and
reimbursement of orphan medicinal
products (OMPs) in EU
Zamora, B., Maignen, F., O’Neill, P., Mestre-
Ferrandiz, J. and Garau, M.
ISPOR European Congress 2017
This study was funded
by Shire

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Objectives
• To compare the availability of and access to
OMPs in the UK (England, Scotland and Wales),
France, Germany, Italy and Spain
• To compare the speed of access to OMPs in the
selected countries
• months between the marketing authorisation and the
decision to recommendation/reimbursement in each
countries.

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Methods
• Data from the European Medicines Agency’s (EMA) and
DG Health and Food Safety’s websites on medicinal
products with an orphan designation and marketing
authorisation
• Time period covered from 2000 to June 2016, i.e. from
inception of Regulation (EC) No 141/2000, to May 2016
• Data on these OMPs were collected concerning
• Health Technology Assessment (HTA), funding or
commissioning, and/or reimbursement decisions
• Date of publication of these decisions
• Systematic and consistent approach to data extraction

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Results - Orphan designations and
marketing authorisations
• EC has granted 1,360 orphan designations, of which 143 (10.5%) have obtained a
marketing authorisation
• There has been an increase in both orphan designations and central marketing
authorisations
2001-
2005
2006-
2010 2011 2012 2013 2014 2015
2016
(Jan-
May) Total
Number of OMPs
designations 173 355 86 118 124 182 185 137 1,360
Number of OMPs
authorised 22 45 7 12 10 17 20 10 143
Average months
from designation
to authorisation 72.7 54.0 47.1 29.4 19.9 15.6 n/a n/a 54.7

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Results –
Access to
OMPs
across EU
countries

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Notes on the previous slide
* 143 OMPs obtained a marketing authorisation since the implementation of
the EU Regulation on Orphan Medicines (Regulation (EC) No 141/2000)
† OMPs reimbursed refers to Health Technology Assessment (HTA)
recommendations to use or inclusion in reimbursement lists in respective
national health systems.

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Limitations
• Country comparisons need to be interpreted with caution
due to different national regulations and procedures, and
the heterogeneous information publicly available
• There are mechanisms ensuring access to OMPs (e.g.
compassionate programmes or individual patient funding
requests)
• HTA recommendations might not necessarily lead to fast
access/impede the possibility of prescribing OMPs in
practice
• Time to access new OMPs is affected by numerous factors
• Only 5 countries of the 28 EU member states were included
• More research is required to estimate uptake of OMPs in
clinical practice

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Summary
• There is still considerable variation in funding and provision of
OMPs across EU countries
• The 143 OMPs are most widely accessible in Germany and
France
• In England, Italy, Scotland, Spain, and Wales between 30% and
60% of OMPs are reimbursed
• In England, less than 50% of OMPs are routinely funded by the NHS,
with one-third of these recommended by NICE
• In Germany reimbursement is automatically granted to OMPs
• The shortest time from authorisation to a reimbursement
decision is in France and Italy (19 months on average), the
longest is in England and Wales (28.5 months on average)

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Thank you!
To enquire about additional information and analyses, please contact
Martina Garau at mgarau@ohe.org
The slides were based on:
Zamora, B., Maignen, F., O’Neill, P., Mestre-Ferrandiz, J. and Garau,
M. (2017). Comparing Access to Orphan Medicinal Products (OMPs) in
the United Kingdom and other European countries. OHE Consulting.
Available at:
https://www.ohe.org/publications/comparing-access-orphan-
medicinal-products-omps-united-kingdom-and-other-european
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Appendix - Key definitions
• Availability
Possibility that an OMP can be prescribed
within the national health system and
dispensed in pharmacies or hospitals
• Access
Health Technology Assessment (HTA)
recommendations to use or inclusion in
reimbursement lists in national health
systems